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Brain of little 'angel' donated for research after 3yo dies from rare Angelman Syndrome

Brain of little 'angel' donated for research after 3yo dies from rare Angelman Syndrome
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FAST joins with University of Pennsylvania to develop investigational AAV gene therapy for Angelman syndrome

The Foundation for Angelman Syndrome Therapeutics (FAST) announced today that the non-profit organization has entered into an exclusive global collaborative research and development agreement with the University of Pennsylvania to develop an investigational adeno-associated virus (AAV) gene therapy for Angelman syndrome (AS). ....

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Couragene co-founders awarded $40M NIH grant

Couragene co-founders awarded $40M NIH grant
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Large NIH Grant Supports CRISPR-based Gene Therapy Development for Brain Diseases

The two-phase grant will support research into a novel CRISPR-based gene-editing technology and delivery platform for targeting neurogenetic diseases. ....

United States , Julie Wolf , Caihong Qiu , Kimberly Greenberg , Allyson Berent , Yong Hui Jiang , James Dziura , Kathleen Cardinale , Hui Zhang , Nigel Bamford , Jennifer Panagoulias , Jiangbing Zhou , Elizabeth Berry Kravis , Eric Velazquez , Caroline Hendry , Michele Spencer Manzon , James Mcpartland , Common Fund Somatic Cell Genome Editing , Pediatric Neurosciencesf Ast Center , Translational Research , National Institutes Of Health , Yale School Of Medicine , Rare Disease Centers Of Excellence , Foundation For Angelman Syndrome Therapeutics , National Institutes , Yale School ,

NIH awards grant to Yale researchers for developing gene editing platform

The National Institute of Health announced a $40 million grant for researchers to develop a CRISPR-mediated platform for genome editing in the brain and to treat genetic disorders. ....

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